Research Fuels Progress Against Cancer

Research continues to be our best defense against cancer. It improves survival and quality of life for millions of people by spurring the development of new and better ways to prevent, detect, diagnose, treat, and, increasingly, cure some of the more than 200 diseases we call cancer.

This progress against cancer is the result of the dedicated efforts of many people working together as part of the biomedical research community. These stakeholders include patients, survivors, family members and friends. Also included in this community are the clinicians and academic researchers. The biotechnology, pharmaceutical and diagnostic companies play an important role in the development of ways to diagnose, treat, detect, prevent and cure cancers. Policymakers and citizen advocates, advocacy and philanthropic organizations have an important role to play. Without continued investments in biomedical research through funding agencies like NIH and NCI, progress against cancer is in jeopardy.

Although the Foundation does not fund clinical trials directly, we have helped put a spotlight on the need for an increased focus on ET, PV and MF research. Our efforts have seen results. While fewer than 20 clinical trials for MPN treatments were conducted from 1995-2005, there have been over 750 trials since 2005.

Clinical trials give patients a chance to try new medical treatments not yet available in the marketplace. During the course of a trial, data is collected recording all observations related to lab results, the patient’s progress (or lack of progress), effectiveness, and side effects of the treatment. Clinical trials are conducted at many sites but usually they are conducted at specialty clinics or teaching medical centers.

There are 4 types or phases.

Clinical Trial Phases

Phase I
Phase I studies are designed to determine the best dose of a therapy and how humans process it, as well as to identity any potential toxicities. These first-in-human studies can also demonstrate early effectiveness or clinical results.
Phase II

Phase II studies determine continued effectiveness of a therapy in a particular disease or a larger group of patients, in addition to continually monitoring for adverse events or potential toxicities.
Phase III
Phase III studies are large trials designed to determine curative value as compared to standard of care (placebos are rarely used in cancer clinical studies).
Phase IV
Phase IV studies are also known as post-marketing studies. They provide additional effectiveness or “real-world” data on the therapy and are conducted after approval by the FDA.

There are many factors to consider when thinking about joining a clinical trial. There are no guarantees that the treatment being tested will be successful. Travel to a clinical trial site may be difficult and costly. The trial may involve more frequent diagnostic tests, such as a bone marrow biopsies. Participants may be asked to stop taking certain medications that would interfere with trial results. 

Whether a trial is right for you is a personal decision that should be discussed with care-givers and family members. The final decision to enroll in a trial should be considered carefully.

Author: Crowley, William

Bill Crowley has worked with blood cancer patients, family and friends for over 15 years. As the Director of Development, he helps supporters appreciate how their gifts impact MPN research.

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